RIBOZYME AND OMICS
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Oral Presentation on Circular RNA research and two poster presentations on platform outcomesValidation of proprietary RNA platform technology and strengthening of global networksRznomics Inc. (CEO Seong-Wook Lee, KOSDAQ 476830), a biopharmaceutical company specializing in RNA-based gene therapeutics, announced today that it successfully concluded the official presentation of its next-generation Circular RNA and oncology gene therapy platforms at the 29th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting held in Boston, MA.ASGCT is the world's largest gathering for gene and cell therapy, where global biotech experts and industry leaders share breakthrough innovations. At this year's meeting, Rznomics showcased its technological prowess through: An Oral Presentation on polymer-based nanocarriers for the systemic delivery of circRNA specifically targeting immune cells in the spleen. Two Poster Presentations regarding circRNA structural engineering and a novel gene therapy platform designed for the simultaneous expression of anti-cancer genes and immune checkpoint inhibitors (ICIs).Rznomics' next-generation circRNA platform garnered significant attention from both academia and industry. A key advantage of the company's polymer-based nanocarrier—distinguished from conventional Lipid Nanoparticles (LNPs)—is the ability to precisely control targeting specificity simply by adjusting the RNA-to-polymer ratio. In an oral presentation based on joint research with Inha University, Rznomics revealed the world's first case of spleen-specific immune cell delivery of circRNA using these carriers. The data demonstrated a three-fold increase in delivery efficiency and a two-fold increase in expression durability compared to linear RNA. These results significantly bolster the feasibility of developing "in vivo CAR-T" therapies, which generate CAR-T cells directly within the patient’s body.In the poster sessions, Rznomics shared its circRNA structural engineering breakthroughs. While the production efficiency of circRNA typically drops sharply as its length increases, Rznomics utilized its proprietary trans-splicing ribozyme engineering to double the production efficiency of long circRNA (over 8kb) compared to existing technologies. This advancement is expected to enhance the mass production and commercial viability of next-generation circRNA therapeutics.The company also introduced preclinical data for RZ-002, a novel oncology gene therapy platform for brain cancer. This technology utilizes the trans-splicing ribozyme to simultaneously express therapeutic genes and ICIs specifically within cancer cells. By utilizing localized expression, RZ-002 demonstrates the potential to overcome the systemic side effects of conventional ICIs and the challenges of penetrating the Blood-Brain Barrier (BBB). The study highlighted the platform's scalability, as it can be adapted for use with various types of ICIs."Through ASGCT, we shared the global competitiveness and scalability of Rznomics' platform technologies with researchers worldwide," said Seong-Wook Lee, CEO of Rznomics. "During the conference, we engaged in in-depth discussions with numerous potential partners regarding strategic collaborations."
RMAT designation based on promising Phase 1b/2a clinical data, including safety profile and preliminary response rates, with trans-splicing ribozyme-based RNA editing platformSEOUL, South Korea, May 8, 2026 /PRNewswire/ -- Rznomics a biopharmaceutical company specializing in RNA-based gene therapeutics, announced today that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) Designation to RZ-001, its lead investigational candidate for the treatment of hepatocellular carcinoma (HCC).RMAT designation is a specialized FDA program created to accelerate the development and review of promising new therapies, including gene therapies, intended to treat serious or life-threatening conditions. Applicant is required to submit preliminary clinical evidence suggesting the potential to address unmet medical needs. This designation provides important opportunities during the drug development process, including increased FDA guidance and eligibility for priority and rolling reviews, as well as accelerated approval pathways. By streamlining these regulatory milestones, the program aims to bring transformative innovations to patients more quickly.RZ-001 is the next-generation oncology therapeutics based on Rznomics' proprietary trans-splicing ribozyme technology platform. By replacing cancer-specific RNA with therapeutic RNA, RZ-001 offers a novel mechanism of action designed to overcome the limitations of conventional therapies. The platform's dual-action approach—enhancing both tumor selectivity and safety—presents a promising new option for HCC patients with limited treatment alternatives. RZ-001 previously received Orphan Drug Designation (ODD) in 2024 and Fast Track Designation (FTD) in 2025 for the treatment of HCC.The FDA's decision to grant RMAT status highlights the clinical potential and innovativeness of RZ-001, particularly following the meaningful interim signals from the Phase 1b/2a clinical trial presented at the American Association for Cancer Research (AACR) Annual Meeting in April 2026."With the RMAT designation, we plan to accelerate our U.S. development and partnership initiatives by initiating formal discussions with the FDA regarding clinical trial design, Chemistry, Manufacturing, and Controls (CMC), and commercialization strategies," said Sung-woo Hong, Vice President of Rznomics.Seong-Wook Lee, CEO of Rznomics, added, "Receiving RMAT designation for RZ-001 is a profound validation of the innovation and competitiveness of our RNA editing platform by the FDA. We will concentrate our resources on global development and commercialization to provide a breakthrough therapeutic option in the field of HCC, where unmet medical needs remain exceptionally high."About RMAT DesignationIntroduced under the 21st Century Cures Act in 2016, the RMAT designation was established to foster the development of innovative regenerative medicine therapies and expand patient access. The program encompasses cell and gene therapies, therapeutic tissue engineering products, and combination products. To be eligible, a drug must be a regenerative medicine therapy intended to treat serious conditions, with preliminary clinical evidence indicating that the drug has the potential to address unmet medical needs for such a condition.
SEOUL, South Korea, May 4, 2026 – Rznomics Inc. (CEO Seong-Wook Lee, KOSDAQ 476830), a biopharmaceutical company specializing in RNA-based gene therapeutics, announced today its participation in the 29th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, to be held in Boston from May 11 to 15.Rznomics will present a total of three abstracts, including one oral presentation and two poster presentations. The company aims to showcase its latest research milestones in proprietary circular RNA (circRNA) technology and its novel oncology gene therapy platforms.ASGCT is the world’s largest professional organization for gene and cell therapy. Its annual meeting serves as a premier global stage for sharing innovative technologies and clinical outcomes. The event continues to grow in scale, with over 8,000 registrants and 2,000 abstracts submitted in 2025.In the oral presentation, Rznomics will introduce its research on polymeric nanocarriers designed for the selective systemic delivery of circular RNA to splenic immune cells. Rznomics’ circRNA is characterized by its high stability, sustained expression, and the absence of residual exogenous sequences. Notably, this technology is gaining significant attention in the industry as a potential in vivo CAR-T platform, capable of generating CAR-T cells directly within the body.The poster presentations will highlight:RNA Structure Engineering: A study on self-targeting and splicing (STS) reactions that enable the efficient circularization of long-form RNAs, which are traditionally difficult to circularize.Next-Gen Oncology Platform: A novel strategy using RNA trans-splicing ribozyme technology. This platform achieves a synergistic therapeutic effect by concurrently expressing therapeutic genes and immune checkpoint inhibitors (ICIs) specifically within cancer cells.“Presenting our latest research at ASGCT, where world-class researchers in gene and cell therapy gather, is a significant milestone,” said Seong-Wook Lee, CEO of Rznomics. “We look forward to demonstrating the differentiation and scalability of our circRNA and trans-splicing ribozyme platform on the international stage, further solidifying our competitive edge in the next-generation RNA therapeutics market.”<ASGCT 2026 Presentation Details>In accordance with ASGCT embargo policies, oral presentation materials will be released at 6:00 AM ET on the day of the presentation, and posters will be available at 6:00 AM ET on May 12, 2026.1. Oral PresentationTitle: Intravenous Delivery of Circular RNAs to Splenic Immune Cells Using Polyaspartamide-Based PolyplexesAbstract Number: 336Date & Time: May 14, 2026; 11:15 – 11:30 AM ETLocation: MCEC Room 206AB (Level 2)2. Poster PresentationTitle: Engineering of P1 Construct for Efficient Circular RNA Generation by End-to-End Self-Targeting and Splicing (STS) Reaction for Long circRNAsAbstract Number: 2122Date & Time: May 13, 2026; 5:00 – 6:30 PM ETLocation: Poster Hall3. Poster PresentationTitle: TERT mRNA Reprogramming for Dual Cancer Therapy: Concurrent Expression of HSVtk and Immune Checkpoint Inhibitor via Trans-Splicing RibozymeAbstract Number: 3317Date & Time: May 14, 2026; 5:00 – 6:30 PM ETLocation: Poster Hall
– RECIST-based
ORR of 38.5% (confirmed) and 46.2% (unconfirmed)
– mRECIST-based
ORR of 61.5% with a complete response (CR) rate of 23%
– No Grade 3 or
higher adverse events related to RZ-001 observed
– Highlights
clinical potential of RNA trans-splicing ribozyme platform technology
Rznomics
announced today that it presented interim clinical data from its ongoing study
of RZ-001, an RNA editing-based investigational anticancer therapy,
in patients with hepatocellular carcinoma (HCC) during an oral presentation at
the AACR 2026 (American Association for Cancer Research Annual Meeting), held
on April 19, 2026, in San Diego, California.
The presentation
was delivered by Professor Yoon-Jun Kim of the Department of Gastroenterology
at Seoul National University Hospital.
The study
includes patients with HCC who are refractory to or ineligible for
transarterial chemoembolization (TACE) and have not received prior systemic
therapy.
According to the
data presented, combination treatment of RZ-001 with atezolizumab and
bevacizumab demonstrated an objective response rate (ORR) of 38.5% (confirmed)
and 46.2% (unconfirmed) based on RECIST v1.1 criteria.
Under mRECIST
criteria, the ORR reached 61.5%, with a complete response (CR) rate of 23%,
suggesting deep tumor responses. Given that mRECIST reflects tumor necrosis,
these findings may indicate meaningful therapeutic impact in HCC.
At interim
analysis, responses are typically categorized as “confirmed” or “unconfirmed.”
Confirmed responses are those validated through subsequent assessments, while
unconfirmed responses represent initial observations. In this study, patients
classified as having “unconfirmed PR” under RECIST criteria had achieved an
initial tumor size reduction of at least 30%.
In terms of
safety, a total of five Grade 3 or higher adverse events were reported as
related to combination agents, including hypertension (2 cases), proteinuria,
hyperglycemia, and gastrointestinal bleeding. Notably, no Grade 3 or higher
adverse events were attributed to RZ-001.
The company
believes these results demonstrate a favorable safety profile for RZ-001, with
no treatment-related safety concerns identified to date, along with encouraging
signals of antitumor activity in terms of both response depth and overall
response rate in combination with standard immunotherapy.
“This oral
presentation at AACR represents an important milestone for our lead program
RZ-001,” said Seong-Wook Lee, Chief Executive Officer of Rznomics. “We are
encouraged by the early efficacy and safety signals observed, and we believe
these data support the potential of RZ-001 as a novel therapeutic approach for
HCC.”
He added, “Beyond
the RZ-001 program, these findings could further support the clinical applicability of our RNA trans-splicing
ribozyme platform technology.”
Hepatocellular Carcinoma (HCC)Hepatocellular Carcinoma (HCC) is the most common type of primary liver cancer, accounting for the vast majority of liver cancer cases worldwide. It originates in the hepatocytes, the main cells of the liver, and is often associated with chronic liver diseases such as cirrhosis or viral hepatitis (B or C). HCC is known for its aggressive nature and high recurrence rates, presenting a significant global health challenge. Because it is often diagnosed at advanced stages, there is a critical unmet medical need for innovative therapies that can provide deeper and more durable responses than current systemic treatments.Transarterial Chemoembolization (TACE)Transarterial Chemoembolization (TACE) is a minimally invasive, localized procedure frequently used to treat patients with intermediate-stage liver cancer. The procedure involves delivering a concentrated dose of chemotherapy directly to the tumor through the hepatic artery, while simultaneously blocking (embolizing) the blood vessels that supply the tumor with oxygen and nutrients. While TACE can be effective for localized control, patients are considered "TACE refractory" when the tumor continues to progress or recurs despite multiple sessions, at which point systemic therapy is typically required.RECIST v1.1 (Response Evaluation Criteria in Solid Tumors)RECIST v1.1 is the standardized international guideline used to evaluate how solid tumors respond to treatment based on anatomical changes in tumor size. It primarily focuses on the sum of the longest diameters of target lesions, where a significant reduction in physical dimensions is categorized as a "response." While it remains the gold standard for most oncology trials, it mainly tracks physical shrinkage and may not fully capture the immediate biological impact of therapies that induce cell death without an immediate change in the overall tumor mass.mRECIST (modified RECIST)mRECIST is a specialized assessment tool developed specifically for Hepatocellular Carcinoma (HCC) to provide a more accurate evaluation of therapeutic efficacy. Unlike traditional criteria that measure the total size of a tumor, mRECIST focuses on the "viable" or living portion by measuring arterial enhancement—the area with active blood flow as seen on imaging. This is particularly critical in HCC because effective targeted or local treatments often cause internal tumor necrosis (cell death). In such cases, the tumor's overall size may remain the same even though the cancer cells are dead, a successful response that mRECIST is uniquely designed to capture.About Rznomics Inc.Rznomics is a clinical-stage biopharmaceutical company based in South Korea focused on developing RNA-based gene therapies. The company's proprietary trans-splicing ribozyme platform enables precise RNA editing and has broad applicability across multiple indications. The company signed a research collaboration and license agreement with global pharmaceutical company Eli Lilly in May 2025 for the development of a novel RNA editing therapeutic and listed on the KOSDAQ market in December 2025. (KOSDAQ 476830)
Rznomics Inc., a biotechnology company specializing in RNA-based gene therapeutics, announced that it will deliver an oral presentation on the interim Phase 1/2a clinical results for ‘RZ-001’—an RNA-based oncology candidate targeting hepatocellular carcinoma (HCC)—at the AACR Annual Meeting 2026, commencing on April 17 (local time). Following the expiration of the official embargo, the clinical abstract has been released.The presentation is based on data from an ongoing clinical trial involving systemic treatment-naive HCC patients who are refractory to or ineligible for Transarterial Chemoembolization (TACE).According to the disclosed abstract, the combination therapy of RZ-001 with atezolizumab and bevacizumab achieved an Objective Response Rate (ORR) of 38.5% based on RECIST v1.1 and 53.8% based on mRECIST. Notably, the Complete Response (CR) rate reached 23% (mRECIST). These figures represent a significant improvement over the IMbrave150 study—the pivotal trial for the current first-line standard of care (atezolizumab/bevacizumab)—showing an ORR increase of over 10% and a CR rate more than double that of the previous landmark study.In HCC clinical evaluations, mRECIST (modified RECIST) is a critical metric alongside RECIST, as it assesses "viable tumor" activity. Since tumors may exhibit internal necrosis (loss of activity) despite limited size reduction, mRECIST provides a more precise evaluation of therapeutic efficacy."Due to the interval between the abstract submission and the conference, we will present even more updated clinical data during our oral session on April 19," said Sung-woo Hong, Vice President of Rznomics. "While these interim results are highly encouraging, we remain cautious and advise against direct comparison with other clinical trials or overinterpretation at this stage."RZ-001 has been granted Orphan Drug Designation (ODD) by the U.S. FDA. This status provides strategic advantages for global commercialization, including clinical trial tax credits, waivers of FDA user fees, and seven years of market exclusivity upon approval.
Rznomics Inc, a RNA-based gene therapeutics leading company, announced that its joint research with Professor Hyun Jin Kim’s team at Inha University has been published in a prestigious international journal. The study focuses on a novel RNA delivery strategy capable of directly targeting immune cells within the body (in vivo). The research, titled “Delivery of Circular RNAs into Splenic Immune Cells via Intravenous Administration of Polyaspartamide Derivative Polyplexes,” was published online on March 27, 2026, in ACS Biomaterials Science & Engineering, a global journal published by the American Chemical Society.This study evaluated the potential of delivering circular RNA (circRNA) to the spleen—an organ rich in immune cells—through simple intravenous (IV) administration. The approach combines Rznomics’ proprietary circular RNA technology with Inha University’s polyamino acid-based polymer carriers.While conventional CAR-T therapies have shown revolutionary success in treating hematologic malignancies by genetically re-engineering a patient’s immune cells ex vivo, they are hindered by complex manufacturing processes, arduous administration protocols, and high costs. Consequently, the ‘in vivo CAR-T’ approach—which directly delivers CAR genes to immune cells inside the patient—is emerging as a next-generation strategy for oncology and autoimmune diseases due to its potential to simplify manufacturing, reduce costs, and significantly improve patient access.Rznomics’ circular RNA features a "clean structure" devoid of unnecessary exogenous sequences, providing superior stability and sustained expression. When paired with Professor Kim’s cationic polymer carrier, which enhances endosomal escape and particle stability, the intracellular delivery efficiency of the RNA is maximized.The research results demonstrated that the Rznomics circular RNA-polymer complex was delivered to various immune cells with exceptional efficiency, showing high selectivity for the spleen upon IV administration. Notably, delivery to the spleen was 10 times higher than to the liver. Furthermore, the circular RNA exhibited twice the half-life and three times the T-cell delivery efficiency in splenic tissue compared to conventional linear RNA. These findings suggest a viable path for creating next-generation CAR-T cell therapies without the need for external cell manipulation."Based on this technology, we will continue to explore the potential applications of polyamino acid polymer-based gene therapies through follow-up studies," said Professor Hyun Jin Kim.Dr. Seong-Wook Lee, CEO of Rznomics commented, "This industry-academic collaboration confirms that our circular RNA technology can be effectively applied to target-specific gene therapies for immune cells in vivo. We plan to continuously expand the scope of our applications to include CAR-T and other advanced therapeutic modalities."This research was supported by the Bio & Medical Technology Development Program under the National Research Foundation of Korea (NRF) and the Basic Research Laboratory Support Program.
Rznomics (CEO Seong-Wook Lee, KOSDAQ 476830), a biotechnology company specializing in the development of RNA-based gene therapies, has announced the title of its oral presentation abstract for the American Association for Cancer Research (AACR) 2026, held in San Diego from April 17 to 22.The presentation is titled: "A phase 1b/2a open-label, multicenter, randomized, dose escalation study evaluating the safety, tolerability and efficacy of RZ-001 in combination with valganciclovir (VGCV) and atezolizumab/bevacizumab in subjects with hepatocellular carcinoma."The presentation, scheduled for April 19, will be delivered by Professor Yoon Jun Kim from the Department of Gastroenterology at Seoul National University Hospital. It will unveil interim safety and efficacy results from the ongoing clinical trial of RZ-001 for the treatment of hepatocellular carcinoma (HCC).A representative from Rznomics stated, "While the interim results of RZ-001 are a major highlight of this AACR presentation, the more significant implication lies in the clinical validation of our platform. This serves as a critical testbed to secure Clinical Proof of Concept (PoC) for Rznomics' proprietary Trans-splicing Ribozyme technology, demonstrating its potential beyond a single pipeline."The flagship pipeline, RZ-001, is a multi-mechanistic anticancer gene therapy that targets telomerase mRNA, which is expressed in over 80% of all cancer types. It is designed to induce cancer cell apoptosis, promote immune cell infiltration, and enhance response rates to immunotherapies. RZ-001 has received Investigational New Drug (IND) approval from the South Korean Ministry of Food and Drug Safety (MFDS) and the U.S. Food and Drug Administration (FDA). Furthermore, it has been granted 'Fast Track' and 'Orphan Drug Designation (ODD)' by the U.S. FDA for both primary liver cancer (HCC) and glioblastoma (GBM).Currently, Rznomics is conducting a Phase 1b/2a clinical trial evaluating RZ-001 in combination with immunotherapy for HCC patients, with patient recruitment and dosing underway across nine institutions in South Korea. The combination agents, Atezolizumab and Bevacizumab, are the primary first-line treatments used in over 70% of advanced HCC cases. However, given that the standard of care (SoC) response rate remains at approximately 20-30%. Rznomics expects significant commercial synergy if the data supports improved clinical efficacy and response rates through the RZ-001 combination.Moreover, since RZ-001 targets telomerase mRNA—prevalent in the majority of malignancies—it holds immense potential for indication expansion across a wide array of solid tumors.
Rznomics Inc. (CEO Seong-Wook Lee) held an Investor Relations (IR) conference on the 3rd December, attended by the CEO and key executives, to share its strategic roadmap and vision following its upcoming listing on the KOSDAQ market.Founded in 2017, Rznomics specializes in developing innovative therapies for cancer and rare intractable diseases using its proprietary RNA replacement enzyme-based RNA editing and correction platform. This platform is uniquely capable of performing three functions—targeting RNA cleavage, splicing, and replacement—within a single therapeutic agent. The technology is garnering significant attention from global pharmaceutical companies and academia due to its scalability across various mutations, its safety, and its optimized delivery efficiency tailored to specific indications.The company’s lead pipeline asset, RZ-001, targets hepatocellular carcinoma (HCC) and glioblastoma (GBM). Both indications have received Orphan Drug Designation (ODD) and Fast Track Designation from the U.S. FDA, raising expectations for early commercialization. For glioblastoma, the therapy has been approved for an Expanded Access Program (EAP) and is currently being administered to patients. For hepatocellular carcinoma, Rznomics is conducting clinical trials in combination with immune-checkpoint inhibitors through clinical collaborations with Roche and Celltrion, which provide the necessary therapeutic agents for the trials free of charge.Beyond RZ-001, the company maintains a diverse pipeline addressing high unmet medical needs, including RZ-003 for Alzheimer’s disease and RZ-004 for retinitis pigmentosa (RP), aiming to provide fundamental solutions for these conditions.Rznomics’ technological benefit has been recognized both domestically and globally. Last year, the company was designated as the first "National Strategic Technology Company" and a "National Strategic Technology Possessing and Managing Company" by the Ministry of Science and ICT (MSIT) and the Korea Institute of Science & Technology Evaluation and Planning (KISTEP). Based on these credentials, Rznomics is pursuing the newly established "Deep Tech Special Listing". Upon listing, it is poised to become the first company among those managed by the MSIT to list under this specialized track.In May, Rznomics validated its technological competitiveness and commercial potential by signing a strategic global licensing agreement with Eli Lilly and Company, valued at approximately KRW 1.9 trillion (USD 1.3 billion), to co-develop RNA editing therapies. This partnership is significant as it secures a first-mover advantage in the next-generation gene therapy market with proprietary RNA editing technology.Rznomics plans to utilize the funds raised through its KOSDAQ listing to: advancing its RNA editing platform & accelerating global clinical trials and commercialization of core pipeline assets. Given the high scalability of its technology platform across various diseases, the company intends to expand collaborations with global pharmaceutical leaders through licensing and joint development based on its original patents and core technologies."Rznomics is presenting a new paradigm in the market for intractable cancer and rare disease treatments, where unmet medical needs are high, based on the world's first Trans-splicing Ribozyme platform," said Dr. Seong-Wook Lee, CEO of Rznomics. "We are committed to establishing our technology as the next-generation standard of care and evolving into a global leader in gene therapy, offering hope to patients suffering from intractable diseases."Rznomics is offering 2,060,000 shares for public subscription. The indicative price range is set between KRW 17,000 and KRW 22,500, with a target fundraising amount of KRW 35.0 billion to KRW 46.4 billion. Demand forecasting for institutional investors will take place from November 27 to December 3, followed by public subscription on December 9 and 10. Samsung Securities and NH Investment & Securities are serving as the joint underwriters.
Rznomics Inc. announced on the 3rd November that it will officially participate in BIO-Europe 2025, a premier global biopharma conference held in Vienna, Austria, from November 3 to 5. BIO-Europe is the largest partnering event in Europe, bringing together over 5,000 industry professionals from more than 60 countries.During the event, Rznomics plans to showcase its proprietary RNA editing platform and gene therapy development strategies. The company will actively seek opportunities for licensing agreements and co-development collaborations through strategic partnering meetings with global pharmaceutical companies, biotech firms, and investors.Rznomics has garnered significant attention within international academic and industrial circles for its differentiated technological platform: "Trans-splicing Ribozyme-based RNA Editing Therapeutics." In May, the company proved its technological innovation and global competitiveness by signing a research collaboration and licensing agreement with the U.S. pharmaceutical giant Eli Lilly and Company, with a total deal value reaching approximately KRW 1.9 trillion (USD 1.3 billion).As global interest in RNA-based therapeutic technologies continues to rise, Rznomics intends to discuss various collaboration models, including platform-based out-licensing and joint R&D, while further refining its plans for global market expansion through these high-level meetings."Since our major licensing deal with Eli Lilly, interest from leading global companies has surged significantly," said Sungwoo Hong, Vice President of Rznomics, who is attending the event. "Our participation in BIO-Europe 2025 will serve as a catalyst to accelerate additional licensing deals and expand our collaborative network with global industry leaders."